RU2012123145A - Генный конструкт (варианты), вектор и рекомбинантный вирусный геном на его основе, вирион, их фармацевтическая композиция, способ in vitro экспрессии полинуклеотида в клетке печеночной природы, лекарственное средство, способ лечения заболевания печени (варианты), индуцируемый двунаправленный оператор-промотор - Google Patents
Генный конструкт (варианты), вектор и рекомбинантный вирусный геном на его основе, вирион, их фармацевтическая композиция, способ in vitro экспрессии полинуклеотида в клетке печеночной природы, лекарственное средство, способ лечения заболевания печени (варианты), индуцируемый двунаправленный оператор-промотор Download PDFInfo
- Publication number
- RU2012123145A RU2012123145A RU2012123145/10A RU2012123145A RU2012123145A RU 2012123145 A RU2012123145 A RU 2012123145A RU 2012123145/10 A RU2012123145/10 A RU 2012123145/10A RU 2012123145 A RU2012123145 A RU 2012123145A RU 2012123145 A RU2012123145 A RU 2012123145A
- Authority
- RU
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- Prior art keywords
- promoter
- sequence
- hepatospecific
- construct
- operator
- Prior art date
Links
- 102000040430 polynucleotide Human genes 0.000 title claims abstract 16
- 108091033319 polynucleotide Proteins 0.000 title claims abstract 16
- 239000002157 polynucleotide Substances 0.000 title claims abstract 16
- 108090000623 proteins and genes Proteins 0.000 title claims abstract 11
- 210000002845 virion Anatomy 0.000 title claims 8
- 239000008194 pharmaceutical composition Substances 0.000 title claims 5
- 230000002457 bidirectional effect Effects 0.000 title claims 3
- 238000000034 method Methods 0.000 title claims 3
- 239000003814 drug Substances 0.000 title claims 2
- 238000000338 in vitro Methods 0.000 title claims 2
- 208000019423 liver disease Diseases 0.000 title claims 2
- 230000002440 hepatic effect Effects 0.000 title 1
- 239000013603 viral vector Substances 0.000 title 1
- 230000001939 inductive effect Effects 0.000 claims abstract 32
- 108091023040 Transcription factor Proteins 0.000 claims abstract 17
- 102000040945 Transcription factor Human genes 0.000 claims abstract 17
- 230000008488 polyadenylation Effects 0.000 claims abstract 13
- 239000004098 Tetracycline Substances 0.000 claims abstract 11
- 229960002180 tetracycline Drugs 0.000 claims abstract 11
- 229930101283 tetracycline Natural products 0.000 claims abstract 11
- 235000019364 tetracycline Nutrition 0.000 claims abstract 11
- 150000003522 tetracyclines Chemical class 0.000 claims abstract 11
- 239000002773 nucleotide Substances 0.000 claims abstract 6
- 125000003729 nucleotide group Chemical group 0.000 claims abstract 6
- 230000000694 effects Effects 0.000 claims abstract 4
- 230000004913 activation Effects 0.000 claims abstract 3
- 230000006698 induction Effects 0.000 claims abstract 2
- 230000003612 virological effect Effects 0.000 claims 7
- 108010088751 Albumins Proteins 0.000 claims 6
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 4
- 210000004027 cell Anatomy 0.000 claims 4
- 102000013462 Interleukin-12 Human genes 0.000 claims 3
- 108010065805 Interleukin-12 Proteins 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 229940117681 interleukin-12 Drugs 0.000 claims 3
- 150000007523 nucleic acids Chemical group 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 201000007270 liver cancer Diseases 0.000 claims 1
- 210000005229 liver cell Anatomy 0.000 claims 1
- 208000014018 liver neoplasm Diseases 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/635—Externally inducible repressor mediated regulation of gene expression, e.g. tetR inducible by tetracyline
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
- C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
- C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| ESP200902122 | 2009-11-05 | ||
| ES200902122 | 2009-11-05 | ||
| PCT/ES2010/070715 WO2011054994A1 (es) | 2009-11-05 | 2010-11-04 | Sistemas de expresión regulada |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2012123145A true RU2012123145A (ru) | 2013-12-10 |
Family
ID=43569271
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2012123145/10A RU2012123145A (ru) | 2009-11-05 | 2010-11-04 | Генный конструкт (варианты), вектор и рекомбинантный вирусный геном на его основе, вирион, их фармацевтическая композиция, способ in vitro экспрессии полинуклеотида в клетке печеночной природы, лекарственное средство, способ лечения заболевания печени (варианты), индуцируемый двунаправленный оператор-промотор |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20120225933A1 (es) |
| EP (1) | EP2497830A1 (es) |
| JP (1) | JP2014503173A (es) |
| CN (1) | CN102712933A (es) |
| AU (1) | AU2010316996A1 (es) |
| BR (1) | BR112012010755A2 (es) |
| CA (1) | CA2780671A1 (es) |
| MX (1) | MX2012005340A (es) |
| RU (1) | RU2012123145A (es) |
| WO (1) | WO2011054994A1 (es) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6591956B2 (ja) * | 2013-03-15 | 2019-10-16 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｍｐｓ１を治療するための組成物および方法 |
| CN105316352A (zh) * | 2014-07-29 | 2016-02-10 | 深圳先进技术研究院 | 一种调节性表达目的基因的载体及其制备方法和应用 |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| EP3184548A1 (en) * | 2015-12-23 | 2017-06-28 | Miltenyi Biotec GmbH | Chimeric antigen receptor with cytokine receptor activating or blocking domain |
| US11208669B2 (en) | 2016-01-12 | 2021-12-28 | University Of Cincinnati | Lentivirus packaging system comprising a synthetic positive feedback loop |
| AU2018298133A1 (en) | 2017-07-06 | 2020-01-23 | The Trustees Of The University Of Pennsylvania | AAV9-mediated gene therapy for treating mucopolysaccharidosis type I |
| CN107365785B (zh) * | 2017-09-11 | 2020-02-18 | 东南大学 | 一种调控细胞内NF-κB活性的基因表达载体及其调控方法和应用 |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| WO2023178067A1 (en) * | 2022-03-16 | 2023-09-21 | Rutgers, The State University Of New Jersey | Controlled muscle‐specific gene delivery |
| CN114891829B (zh) * | 2022-05-24 | 2023-09-26 | 新乡医学院 | 一种肝特异性游离型表达载体和基因治疗载体及其应用 |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4970154A (en) | 1987-10-09 | 1990-11-13 | Baylor College Of Medicine | Method for inserting foreign genes into cells using pulsed radiofrequency |
| US5676954A (en) | 1989-11-03 | 1997-10-14 | Vanderbilt University | Method of in vivo delivery of functioning foreign genes |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| EP0728214B1 (en) | 1993-11-09 | 2004-07-28 | Medical College Of Ohio | Stable cell lines capable of expressing the adeno-associated virus replication gene |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5484720A (en) | 1994-09-08 | 1996-01-16 | Genentech, Inc. | Methods for calcium phosphate transfection |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| WO1996017947A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| US5891680A (en) | 1995-02-08 | 1999-04-06 | Whitehead Institute For Biomedical Research | Bioactive fusion proteins comprising the p35 and p40 subunits of IL-12 |
| CA2222550A1 (en) | 1995-06-07 | 1996-12-19 | Baylor College Of Medicine | Nucleic acid transporters for delivery of nucleic acids into a cell |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| US5679559A (en) | 1996-07-03 | 1997-10-21 | University Of Utah Research Foundation | Cationic polymer and lipoprotein-containing system for gene delivery |
| AU4645697A (en) | 1996-09-11 | 1998-04-02 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Aav4 vector and uses thereof |
| JP2001506132A (ja) | 1996-12-18 | 2001-05-15 | ターゲティッド ジェネティクス コーポレイション | Ａａｖベクターの産生における使用のためのリコンビナーゼ活性化可能ａａｖパッケージングカセット |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| JP4060531B2 (ja) | 1998-05-28 | 2008-03-12 | アメリカ合衆国 | Ａａｖ５ベクターおよびその使用 |
| AU763183B2 (en) * | 1998-08-18 | 2003-07-17 | Research Development Foundation | Methods to enhance and confine expression of genes |
| US6103526A (en) | 1998-10-08 | 2000-08-15 | Protein Sciences Corporation | Spodoptera frugiperda single cell suspension cell line in serum-free media, methods of producing and using |
| JP4573437B2 (ja) | 1998-11-05 | 2010-11-04 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス血清型１核酸配列、ベクターおよび同一物を含有する宿主細胞 |
| CA2351015C (fr) * | 1998-11-09 | 2011-09-27 | Aventis Pharma S.A. | Nouveau systeme de regulation de l'expression d'un transgene |
| CA2348382C (en) | 1998-11-10 | 2013-09-17 | The University Of North Carolina At Chapel Hill | Chimeric parvovirus vectors and methods of making and administering the same |
| ES2146552B1 (es) | 1998-11-24 | 2001-04-16 | Inst Cientifico Tecnol Navarra | Peptidos inhibidores de tgf/31 |
| US6387368B1 (en) | 1999-02-08 | 2002-05-14 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| EP1317288A2 (en) * | 2000-09-11 | 2003-06-11 | MUSC Foundation For Research Development | Method and composition for treating tumors by selective induction of apoptosis |
| US6835568B2 (en) * | 2001-10-30 | 2004-12-28 | Virxsys Corporation | Regulated nucleic acid expression system |
| US7271002B2 (en) | 2001-11-09 | 2007-09-18 | United States Of America, Represented By The Secretary, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| AU2003234734A1 (en) | 2002-04-29 | 2003-11-17 | Saint Louis University | Peptide antagonists of tgf-beta family members and therapeutic uses thereof |
| DK3235827T3 (da) | 2003-06-19 | 2021-04-19 | Genzyme Corp | Aav-virioner med reduceret immunreaktivitet og anvendelser deraf |
| ES2304069B1 (es) | 2003-08-22 | 2009-08-12 | Proyecto De Biomedicina Cima, S.L. | Peptidos con capacidad de unirse al factor transformante de crecimiento beta 1 (tgf-b1). |
| US6852505B1 (en) * | 2003-10-15 | 2005-02-08 | Chau-Ting Yeh | Hepatitis |
| US8242081B2 (en) | 2005-12-06 | 2012-08-14 | Centre National De La Recherche Scientifique | Cell penetrating peptides for intracellular delivery of molecules |
| CN101481704A (zh) * | 2009-01-21 | 2009-07-15 | 中国人民解放军第四军医大学 | 一种四环素严紧调控的真核表达载体 |
-
2010
- 2010-11-04 BR BR112012010755A patent/BR112012010755A2/pt not_active IP Right Cessation
- 2010-11-04 MX MX2012005340A patent/MX2012005340A/es not_active Application Discontinuation
- 2010-11-04 WO PCT/ES2010/070715 patent/WO2011054994A1/es active Application Filing
- 2010-11-04 JP JP2012537425A patent/JP2014503173A/ja not_active Withdrawn
- 2010-11-04 CA CA2780671A patent/CA2780671A1/en not_active Abandoned
- 2010-11-04 US US13/508,494 patent/US20120225933A1/en not_active Abandoned
- 2010-11-04 EP EP10795406A patent/EP2497830A1/en not_active Withdrawn
- 2010-11-04 AU AU2010316996A patent/AU2010316996A1/en not_active Abandoned
- 2010-11-04 RU RU2012123145/10A patent/RU2012123145A/ru not_active Application Discontinuation
- 2010-11-04 CN CN201080060595XA patent/CN102712933A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2011054994A1 (es) | 2011-05-12 |
| BR112012010755A2 (pt) | 2015-09-22 |
| EP2497830A1 (en) | 2012-09-12 |
| AU2010316996A1 (en) | 2012-06-14 |
| CA2780671A1 (en) | 2011-05-12 |
| US20120225933A1 (en) | 2012-09-06 |
| JP2014503173A (ja) | 2014-02-13 |
| CN102712933A (zh) | 2012-10-03 |
| MX2012005340A (es) | 2012-12-05 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA92 | Acknowledgement of application withdrawn (lack of supplementary materials submitted) |
Effective date: 20150311 |